October 2018 Update
Robert Fuhlbrigge, MD, PhD
Children’s Hospital Colorado
They say time flies when you are having fun. Even so, it is hard for me to believe that three months of my tenure as President of CARRA are already behind me. There are many exciting things happening within CARRA, and I wanted to take a moment to highlight just a few of these.
First, I want to congratulate the sites enrolling patients in the Registry for a job well done- As of Oct 2018 we have over 6700 patients enrolled, with the registry collecting data on patients with JIA as well as a selection of SLE and JDM. In addition, we have successfully finishing enrolling the STOP-JIA comparative effectiveness study of the polyarticular JIA Consensus Treatment Plans (CTPs). Localized and systemic scleroderma are on the cusp of initiating enrollment and we are working plans to engage patients with other conditions.
Second, I would like to highlight some of the events of the past few months that have impact on our mission and future activities. These have included:
- New Approaches to Evaluate the Efficacy and Safety of Therapeutics for Juvenile Idiopathic Arthritis (JIA): A CARRA sponsored Discussion: This CARRA organized meeting brought together stakeholders (CARRA, Arthritis Foundation, patient/family advocates and international pediatric rheumatologists) with regulatory agencies (FDA and EMA (European Medicines Agency)) and pharmaceutical industry representatives. The objective of the meeting was to discuss the limitations of current clinical trial designs in fulfilling the need for the timely authorization of new medications for JIA and to propose solutions such innovative clinical trial designs using outcome measures that are more meaningful. The feedback from participants was very positive, and results of this meeting will be submitted for publication in a joint manuscript in the near future.
- Externally-led FDA JIA Patient Focused Drug Development Meeting (PFDD): Jointly organized by the Arthritis Foundation and CARRA, this meeting provided an opportunity for patients, caregivers and other JIA advocates to share their perspectives with the FDA, drug developers, healthcare providers and other stakeholders. Patients and caregivers described the impact of JIA and its treatment(s) on their daily lives, and highlighted the need for improved access to current treatments as well as patient-friendly approaches to investigation/ approval of new therapies. You can view a part of the PFDD conference at https://www.youtube.com/watch?v=8oDhqlfcMJI and a video recording of the entire day will be available soon. Be prepared with tissues as the emotions run high at times and the insights provided were wake-up call to everyone.
- CARRA-Arthritis Foundation Research Coordinator Pilot program: This program, new in 2018, is designed to see if start-up funds to help hire a research coordinator can assist sites in increasing enrollment and data entry in the CARRA Registry. Eight sites were selected and initial results are highly encouraging- showing a 200% increase overall in enrollment over the first 3 months of the program. It helps to have help
- CTPs, Comparative Effectiveness Research and Clinical Trials: In addition to the successful completion of enrollment in the STOP-JIA study, highlights on the research front include: initiation of the anaSTILLs study- a registration study of anakinra in systemic JIA and Still’s Disease; continuing enrollment in the FROST study of the systemic JIA CTPs; close to complete enrollment of the PEPR study validating the PROMIS Patient Reported Outcomes in JIA and SLE; initiation of the PROMOTE study of methotrexate pharmacogenomics; and preparations to launch LIMIT-JIA, a new PCORI funded randomized trial of abatacept to prevent uveitis and extension in oligo-JIA. Go to the CARRA website to see additional exciting news about our research--CARRA is truly moving the bar forward in establishing an evidence base for care of our patients.
These are exciting times at CARRA- our hard work is paying off with progress on many fronts. I hope you will join me in giving kudos to your colleagues who have worked so diligently to make these projects successful.
I am looking forward to seeing everyone who is able to come to the ACR Annual meeting in Chicago. Please join us at the CARRA meeting and the CARRA-AF reception on Monday evening starting at 6:30 PM CT at the Hyatt hotel adjacent to the convention center.
Rob Fuhlbrigge, MD, PhD
President, CARRA Inc.